On May 8, 2017 Kite Pharma, Inc. (NASDAQ:KITE), a cell therapy company, reported first quarter 2017 financial results and provided a corporate update for the period ended March 31, 2017 (Press release, Kite Pharma, MAY 8, 2017, View Source [SID1234518912]).

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"Kite is intensely focused on bringing axicabtagene ciloleucel to market in 2017. Our preparation for the potential commercialization of the first CAR-T therapy in aggressive non-Hodgkin lymphoma began two years ago. With the team and infrastructure we now have in place, we are confident in our readiness to deliver upon potential approval in the U.S. and expect to file for approval in Europe in the third quarter of this year," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer of Kite. "We are also keeping an eye toward future growth with additional indications across the KTE-C19 program and development of earlier stage product candidates, including KITE-585, which we believe has the potential to become the next significant opportunity for Kite."

First Quarter 2017 Financial Results

Revenues were $9.8 million for the first quarter of 2017.
Research and development expenses were $65.9 million for the first quarter of 2017, which includes $12.7 million of non-cash stock-based compensation expense.
General and administrative expenses were $35.8 million for the first quarter of 2017, which includes $11.4 million of non-cash stock-based compensation expense.
Net loss was $90.4 million, or $1.74 per share, for the first quarter of 2017.
Non-GAAP net loss for the first quarter of 2017 was $66.3 million, or $1.28 per share, excluding non-cash stock-based compensation expense of $24.1 million.
As of March 31, 2017, Kite had $804.0 million in cash, cash equivalents, and marketable securities. A public offering of common stock generated approximately $409.7 million in gross proceeds to Kite. In addition, Kite received a $50 million upfront payment related to its strategic collaboration with Daiichi Sankyo.
2017 Financial Guidance

Kite continues to expect the full year 2017 net cash burn to be between $325 million and $340 million. This guidance assumes GAAP operating expenses to be between $490 million and $515 million, which includes approximately $135 million in non-cash stock based compensation expense. Kite expects 2017 total operating expenses to consist of approximately 60 percent Research and Development and approximately 40 percent General and Administrative.
First Quarter 2017 and Recent Highlights

Axicabtagene Ciloleucel/KTE-C19 Progress

Presented positive topline results from the primary analysis of the ZUMA-1 study of axicabtagene ciloleucel in patients with aggressive non-Hodgkin lymphoma (NHL) at the 2017 American Association of Cancer Research annual meeting. Data included the 99 percent success rate in the manufacturing of clinical product patient dose from a single apheresis for the multi-center ZUMA-1 clinical trial.
Completed the submission of a Biologics License Application (BLA) to the FDA for axicabtagene ciloleucel in patients with aggressive NHL.
Initiated ZUMA-5 study of axicabtagene ciloleucel in patients with follicular NHL.
Initiated ZUMA-9 study to provide patients access to axicabtagene ciloleucel during the regulatory review period.
Strategic Collaborations

Entered a strategic collaboration with Daiichi Sankyo to develop and commercialize axicabtagene ciloleucel in Japan.
Established a joint venture with Fosun Pharma to develop and commercialize T-cell therapies, including axicabtagene ciloleucel, in China.
Pipeline Expansion

Cleared an investigational new drug (IND) application for KITE-718, a T cell receptor (TCR) cell therapy candidate that targets MAGE-A3/A6 antigens expressed on solid tumors.
Commercial Preparation

Started recruitment and training of cell therapy account managers to support customer service and logistical coordination.
Additional 2017 Clinical Milestones

KTE-C19 and axicabtagene ciloleucel

Submit marketing authorization application (MAA) to the European Medicines Authority (EMA) for axicabtagene ciloleucel in aggressive NHL in the third quarter of 2017.
Availability of preliminary 12-month follow-up data from ZUMA-1 study of axicabtagene ciloleucel in patients with aggressive NHL.
Availability of preliminary follow-up Phase 1 data from ZUMA-3 and ZUMA-4 studies of pediatric and adult acute lymphoblastic leukemia, respectively.
Advance ZUMA-3 and ZUMA-4 studies into Phase 2.
Availability of preliminary data from ZUMA-6 combination study of axicabtagene ciloleucel and atezolizumab PD-L1 checkpoint inhibitor in aggressive NHL.
Cell Therapy Pipeline

Initiate Phase 1 study of KITE-718 in patients with solid tumors in the second quarter of 2017.
File IND for KITE-585, a CAR T cell therapy candidate that targets BCMA for the treatment of multiple myeloma, in the third quarter of 2017.

On May 8, 2017 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the first quarter ended March 31, 2017 and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, MAY 8, 2017, View Source [SID1234518908]).

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"We had a productive first quarter across our pipeline," said Rick Fair, Bellicum’s President & Chief Executive Officer. "We continued to make progress on the registration trial for BPX-501, and presented updated clinical data highlighting its potential to transform patients’ lives. We are actively recruiting initial clinical trials with our controllable CAR T and TCR product candidates, and presented preclinical data on exciting new enhancements to our pioneering technology platform. This progress underscores our commitment to developing novel cell therapies in areas of dire need."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

BPX-501
Adjunct T-cell therapy, administered after allogeneic hematopoietic stem cell transplantation, to support faster immune recovery, improved infection control, and reduced mortality and Graft versus Host Disease (GvHD)

Registration Studies Advancing in the European Union
Bellicum continues to enroll its registration trial in the E.U. with BPX-501 and rimiducid in pediatric patients with orphan inherited blood disorders or hematologic cancers receiving a haploidentical transplant, and is preparing to initiate a separate observational trial in a comparative sample of patients receiving a matched unrelated donor, or MUD, transplant to support regulatory submission.

Preparation Ongoing for U.S. Registration Trials
Bellicum continues to prepare for pivotal trials of BPX-501 in the U.S. in pediatric patients with orphan inherited blood disorders and blood cancers and in adults with high- and intermediate-risk AML receiving haploidentical transplant.

Data Update Highlights Promise of BPX-501 Clinical Program
At the Bone Marrow Transplant (BMT) Tandem Meeting in February, Bellicum reported data from the BP-004 trial which showed a low incidence of transplant-related mortality, rapid immune recovery, a low rate of GvHD that was manageable with standard treatments or rimiducid, and no serious adverse events associated with the use of BPX-501 or rimiducid.
BPX-601
Novel GoCAR-T product candidate designed with the proprietary iMC activation switch to improve efficacy

Phase 1 BPX-601 Clinical Trial Underway
Bellicum is evaluating its first GoCAR-T product candidate in patients with nonresectable pancreatic cancer who test positive for prostate stem cell antigen (PSCA).
BPX-701
High affinity T-cell receptor (TCR) product candidate designed with the CaspaCIDe safety switch

Phase 1 BPX-701 Clinical Trial Enrolling
The trial is recruiting HLA-A2 positive patients with refractory or relapsed acute myeloid lymphoma (AML) and myelodysplastic syndromes (MDS) who test positive for preferentially-expressed antigen in melanoma (PRAME).
PRECLINICAL RESEARCH

In April, Bellicum reported positive preclinical data at AACR (Free AACR Whitepaper) on the first-ever dual-switch technology incorporated into CAR T and TCR constructs, an approach offering the possibility of both activating cells to enhance efficacy and eliminating them to manage toxicity in a single product.

FIRST QUARTER 2017 FINANCIAL RESULTS

Cash Position and Guidance: During the first quarter of 2017, Bellicum completed an underwritten public offering of common stock that provided approximately $64.6 million of net proceeds. Bellicum also borrowed the final $10.0 million tranche under its loan agreement with Hercules Capital. As of March 31, 2017, the Company had cash, restricted cash and investments totaling $164.6 million, compared to $113.4 million at December 31, 2016. Based on current operating plans, Bellicum expects to end 2017 with approximately $85 to $95 million in cash and investments, and that current cash resources will be sufficient to meet operating requirements through the end of 2018.

Net Loss: Bellicum reported a net loss of $22.0 million for the first quarter of 2017, compared to a net loss of $15.1 million for the first quarter of 2016. The results included non-cash, share-based compensation charges of $3.4 million and $3.1 million for the first quarter of 2017 and 2016, respectively.

R&D Expenses: Research and development expenses were $15.3 million and $10.9 million for the three months ended March 31, 2017 and 2016, respectively. The higher 2017 costs were due primarily to an additional $2.9 million of clinical development expenses for BPX-501 reflecting increased clinical trial activities and manufacturing costs due to increased enrollment in clinical trials, and an additional $1.5 million of expenses for increased personnel, overhead charges and manufacturing facility start-up costs.

G&A Expenses: General and administrative expenses were $5.9 million for the three months ended March 31, 2017, and $4.3 million for the three months ended March 31, 2016. The increase in G&A expenses of $1.6 million in 2017, was due primarily to higher personnel costs as a result of hiring additional employees and to severance costs.

On May 8, 2017 GlycoMimetics, Inc. (NASDAQ: GLYC) reported progress on its clinical development programs and its financial results for the first quarter ended March 31, 2017 (Filing, Q1, GlycoMimetics, 2017, MAY 8, 2017, View Source [SID1234518898]).

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"In the first quarter of 2017, GlycoMimetics delivered on its plan to provide updates on both data and the status of its ongoing pipeline programs. The acceptance of our clinical and preclinical abstracts for presentation at key oncology congresses, including AACR (Free AACR Whitepaper) and more recently, ASCO (Free ASCO Whitepaper), reinforces our enthusiasm for the GMI-1271 and GMI-1359 programs, and demonstrates to the scientific and physician communities the relevance of our work to address unmet medical needs in a novel way. As we look forward to the remainder of 2017, our plan is to build on this progress with further data updates: at the Annual ASCO (Free ASCO Whitepaper) meeting in Chicago followed by the European Hematology Association (EHA) (Free EHA Whitepaper) meeting, both before the end of the second quarter in June. Additionally, we can report that the Phase 3 program to evaluate rivipansel for vaso-occlusive sickle cell crisis, according to our partner Pfizer, remains on track for completion in the second half of 2018, " said Rachel King, GlycoMimetics’ Chief Executive Officer.

Company management will host a conference call on Thursday, May 18, 2017 at 8:30 a.m. Eastern time to provide a clinical data update from the abstracts for the upcoming ASCO (Free ASCO Whitepaper) conference. A question and answer session with the GlycoMimetics team will follow the company’s remarks. The call can be accessed by dialing (844) 413-7154 (U.S. and Canada) or (216) 562-0466 (international) and entering passcode 4110139. To access the live audio webcast, or the subsequent archived recording, visit the "Investors – Events & Presentations" section of the GlycoMimetics website at www.glycomimetics.com. The webcast will be recorded and available for replay on the GlycoMimetics website for 30 days following the call.

Key Operational Highlights for the First Quarter of 2017:
·
The first of two patient cohorts in the Phase 2 portion of the AML trial of GMI-1271 has completed enrollment. This cohort is comprised of 25 patients 60 years of age or older with newly diagnosed AML. The study is designed to evaluate the potential of GMI-1271, GlycoMimetics’ E-selectin antagonist drug candidate, in combination with chemotherapy, as a treatment for patients with both newly diagnosed and relapsed/refractory AML. Enrollment in the study’s second cohort is expected to complete in the middle of this year. The two arms combined will enroll a total of about 90 patients.
·
Pre-clinical research supporting the potential of two of its drug candidates, GMI-1271 and GMI-1359, against multiple myeloma was shared via an oral presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017. Combination therapy of carfilzomib with GMI-1271 or GMI-1359 prolonged survival of mice with multiple myeloma over treatment with carfilzomib alone.
·
The company announced that it will provide an update on clinical data from its Phase 1/2 study of GMI-1271 in AML at the 2017 American Society for Clinical Oncology in Chicago. GMI-1271 is an antagonist of E-selectin, for which prior clinical data has shown an emerging and differentiated potential efficacy and safety profile.


First Quarter 2017 Financial Results:

·

Cash position: As of March 31, 2017, GlycoMimetics had cash and cash equivalents of $34.6 million as compared to $40.0 million as of December 31, 2016. Subsequent to March 31, 2017, the Company has raised an additional $3.8 million in net proceeds under the at-the-market facility.

·

R&D Expenses: The company’s research and development expenses increased to $5.9 million for the quarter ended March 31, 2017 as compared to $5.5 million for the first quarter of 2016. The increase was due to on-going costs associated with the clinical trials for GMI-1271 in AML and MM, partially offset by a decrease in expenses related to manufacturing and process development for GMI-1271.

·

G&A Expenses: The company’s general and administrative expenses remained at $2.1 million for both the quarters ended March 31, 2017 and 2016.

·

Shares Outstanding: Shares outstanding as of March 31, 2017 were 23,855,934.

About GMI-1271
GMI-1271 is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. Preclinical research points to the drug’s potential role in moving cancerous cells out of the protective environment of the bone marrow where they hide and escape the effects of chemotherapy. In preclinical studies using animal models of AML, the results of which were presented at ASH (Free ASH Whitepaper) meetings, GMI-1271 was also associated with a reduction of chemotherapy-induced neutropenia and chemotherapy-induced mucositis.
About GMI-1359
GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules that keep cancer cells in the bone marrow. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and MM.

On May 8, 2017 DelMar Pharmaceuticals (Nasdaq: DMPI) ("DelMar" and the "Company"), a biopharmaceutical company focused on developing new cancer therapies, reported new mechanism of action (MOA) data for its anti-cancer product candidate, VAL-083 (dianhydrogalactitol), a "first-in-class" small-molecule DNA-targeting agent in temozolomide (Temodar)-resistant GBM, at the World Federation of Neuro-Oncology Societies (WFNOS) (Press release, DelMar Pharmaceuticals, MAY 8, 2017, View Source [SID1234518897]).

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The Company presented two posters at the Quadrennial session of the WFNOS Conference in Zurich, Switzerland.

The titles and summaries of the posters are as follows:

Abstract 1: Distinct mechanism of action of dianhydrogalactitol (VAL-083) overcomes chemoresistance in glioblastoma. Zhai et al, 2017.

It has been previously demonstrated, both preclinically and in the clinic, that VAL-083 is active in temozolomide-resistant GBM. Glioblastoma cells are known to become refractory to treatment because they are able to repair the DNA damage caused by temozolomide using the MGMT* enzyme and the MMR** family of enzymes. While temozolomide is inactive in GBM cells where MGMT is functional, it has already been demonstrated that the activity of VAL-083 is unaffected by the MGMT status of a cell and hence VAL-083 remains active, even when temozolomide has failed.

The authors now show that VAL-083 is not only able to circumvent the MGMT pathway but is also able to override the secondary temozolomide resistance mechanism seen in GBM—MMR DNA repair. In cancer cell-lines VAL-083 is able to induce DNA damage and resultant cell apoptosis even when MMR enzymes (MLH-1 and MSH-2) are made constitutively active.

Taken together, these data provide preclinical evidence, and support the clinical rationale, that VAL-083 should be studied as a front-line therapy for GBM patients in whom active MGMT and MMR pathways will result in chemoresistance to temozolomide.

These results also add to the growing evidence that temozolomide may only be a viable front-line treatment in the 1/3 of GBM patients for whom biomarker data reveal inactive MGMT and MMR enzymes. VAL-083, whose activity is independent of, and agnostic to, the MGMT and MMR activity, may in fact be a superior alternative.

Abstract 2: Clinical trials of VAL-083 in patients with chemoresistant glioblastoma. Bacha et al, 2017.

In this poster the authors review historic clinical trials of VAL-083 run by the National Cancer Institute and the modern dose-finding trials conducted by DelMar (ASCO 2016) to provide the rationale for the ongoing and new clinical development of VAL-083 in GBM. This topic was also the recent subject of a contributed editorial by CEO Jeff Bacha and CSO Dennis Brown for Life Science Leader, entitled Cancer Breakthroughs: A Look to the Past Can be a Look to the Future.

DelMar has announced plans to investigate VAL-083 in Phase 2 and Phase 3 clinical trials in recurrent and newly diagnosed GBM. A pivotal randomized, controlled Phase 3 Study in Temozolomide-Avastin Recurrent GBM ("STAR-3") will evaluate the overall survival of VAL-083 versus salvage chemotherapy for GBM patients who have previously failed both temozolomide and bevacizumab (Avastin) and for whom there exists no approved treatment option. Should VAL-083 show a survival benefit in this moribund, recalcitrant population, it could revolutionize the GBM treatment landscape. A Phase 2 study of VAL-083 at MD Anderson Cancer Center is currently enrolling second-line GBM patients who have failed front-line temozolomide and test positive for MGMT. Finally, a Phase 2 trial enrolling MGMT-expressing front-line GBM patients to be treated with VAL-083 in lieu of temozolomide has the potential to introduce biomarker testing into the GBM treatment paradigm to determine which patients will receive temozolomide and VAL-083, respectively, based on their MGMT expression status.

*MGMT= O6-Methyl Guanine DNA-Methyl Transferase **MMR=mismatch repair

About VAL-083

VAL-083 is a "first-in-class," small-molecule chemotherapeutic that demonstrated clinical activity against a range of cancers including GBM in historical clinical trials sponsored by the U.S. National Cancer Institutes (NCI). DelMar has demonstrated that VAL-083’s anti-tumor activity against GBM is unaffected by the expression of MGMT and MMR in vitro. Further details can be found at View Source

VAL-083 has received an orphan drug designation in Europe for the treatment of malignant gliomas and the U.S. FDA Office of Orphan Products has granted an orphan designation to VAL-083 for the treatment of glioma, medulloblastoma and ovarian cancer. Based on historic clinical trials run by the NCI, the modern Phase 1/2 dose finding trial run by DelMar in GBM (ASCO 2016), and recent guidance from the FDA, the Company has embarked on Phase 2 or 3 trials for VAL-083 across recurrent and newly diagnosed GBM. DelMar has announced plans to advance VAL-083 into a pivotal randomized multi-center Phase 3 clinical trial for the treatment of bevacizumab-failed GBM, a Phase 2 trial (with MD Anderson Cancer Center) in first recurrence GBM patients prior to bevacizumab therapy, and into a separate international Phase 2 trial for newly diagnosed MGMT-unmethylated GBM. DelMar believes that data from its clinical trials, if successful, will form the basis of a new treatment paradigm for the vast majority of GBM patients whose tumors exhibit features that make them unlikely to respond to currently available therapies.

About Glioblastoma Multiforme (GBM)

GBM is the most common and aggressive primary brain cancer. Current standard of care includes surgery, radiation and treatment with temozolomide (TMZ), however nearly all tumors recur and the prognosis for recurrent GBM is dismal. Most GBM tumors have unmethylated promoter status for MGMT. Second-line treatment with anti-angiogenic agent bevacizumab has not improved overall survival (OS) and 5-year survival is less than 3%. VAL-083 (dianhydrogalactitol) is a first-in-class bi-functional DNA-targeting agent that induces interstrand DNA cross-links at the N7-position of guanine leading to DNA double-strand breaks and cell death in GBM cell lines and GBM cancer stem cells, independent of MGMT or MMR status in vitro. VAL-083 readily crosses the blood-brain barrier and accumulates in brain tumor tissue. Our recent Phase 1/2 clinical trial in recurrent GBM patients failing both TMZ and bevacizumab, suggested that VAL-083 offers clinically meaningful survival benefits for patients with recurrent GBM and pinpointed a new dosing regimen (40 mg/m2/d on days 1,2,3 of a 21-day cycle) which was well-tolerated and was selected for study in subsequent GBM trials. These trials include, i) an ongoing single-arm, biomarker driven, Phase 2 study to determine if VAL-083 treatment of MGMT-unmethylated adult GBM patients at first recurrence/progression, prior to bevacizumab improves overall survival, compared to historical control with lomustine (clinicaltrials.gov identifier: NCT02717962). ii) A pivotal Phase 3 study in recurrent GBM after failing both TMZ and bevacizumab. The control arm will consist of a limited number of salvage chemotherapies currently used in bevacizumab-failed GBM. If successful, this study will serve as the basis for a New Drug Application (NDA) submission for VAL-083. iii) A single arm, biomarker driven, Phase 2 study to confirm the tolerability and efficacy of VAL-083 in combination with radiotherapy in newly diagnosed MGMT-unmethylated GBM patients whose tumors are known to express high MGMT levels. The results of these studies may support a new treatment paradigm in chemotherapeutic regimens for the treatment of GBM.